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Hope Through Research

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.

Manufacturing: Can You Produce a Consistent Drug in Large Amounts?

This is one of the more mundane stages of drug development, but nonetheless crucial.

You only have to be one of the individuals with Pompe disease who couldn’t get the lifesaving drug Myozyme when its developer, Genzyme, had issues with its manufacturing sites to know that there is not much use in a treatment if there is none available. And no one would want to take a drug that may or may not include toxic ingredients, or a variable amount of the active ingredient.

Thus, even at the preclinical stage, the developer has to be able to show that he or she can produce exactly the same drug substance each and every time, even when they ramp up production to much larger amounts than needed to test it in a few mice.

This sounds simple, and indeed may be simple, in the case of a small molecule produced by a simple chemical process. It is much more complicated for a complex biologic drug like Myozyme, where even tiny changes in the manufacturing process can change the drug. That is why, when Genzyme opened a new plant to make Myozyme, it was treated as a new drug (called Lumizyme), and had to go through approvals again.

Companies must demonstrate that they have a robust protocol to make their drug and the ability to make enough uniform drug for clinical trials.